Gene therapy safety and success proven once again

The news just came in. A third gene therapy trial for Leber Congenital Amaurosis caused by mutations in RPE65, carried out at the University of Florida, has proven safe, and has led to improvement of vision in 4 out of the 6 participants. Only a few days ago, the world of science was shaken by similar results obtained by 2 analogous trials conducted in London and in the US (see previous posts).


More to come for sure!

Message from David Brint, President of FRR

Dear Friends:
As some of you may have heard, Jean Bennett at the Children's Hospital of Philadelphia has announced the initial results of the RPE 65 gene therapy trial. Jean has reported that all three of the injected patients have had some restored vision. This includes vision of up to three lines on an eye chart andimproved ambulatory vision.
This is the most significant result for the restoration of sight in history.
You all should know that the current trial is only a safety trial and has only included a half dose of the gene. What this all means is that the gene vectoris not causing any health problems to date and that the results may improve with increased dosing.
Gene therapy will be a potential treatment for all forms of LCA where photoreceptors are intact.
The FRR has been a supporter of Jean Bennett's work and we are proud of her team's accomplishment.
The attached web links will give you more information
http://abcnews.go.com/Health/wireStory?id=4737873http://content.nejm.org/cgi/content/full/NEJMoa0802268
Sincerely,
David Brint
President FRR

Clinical trial recap

So after the initial chaotic excitement, let us try to recap:
two separate gene therapy trials for RPE65 LCA proved the treatment is safe and can work. One was carried out in England, at Moorfields Eye Hospital and the University College London, the other at the Children’s Hospital of Philadelphia in collaboration with The University of Pennsylvania, the Seconda Università of Naples and the Telethon Institute of Genetics and Medicine, also of Naples - go Italy!
Both trials enrolled three patients. The treatment proved safe for all six patients, and it improved vision in all three subjects in the Italian/US trial and in 1 of the London trial.

Both papers were published in the New England Journal of Medicine on the same day. Here are the links to the articles’ abstracts

London trial

US/Italian trial


Certainly a lot to absorb, but the message is simple: the road to future trials for Leber's Congenital Amaurosis and other retinal genetic disorders is now open!